Academic Chair for Regulation in Law, Medicine, and Technology, Faculty of Law and Faculty of Medicine, University of Zurich, Zurich, Switzerland.
Academic Chair for Regulation in Law, Medicine, and Technology, Faculty of Law and Faculty of Medicine, University of Zurich, Zurich, Switzerland
BMJ Open. 2023 Dec 18;13(12):e078134. doi: 10.1136/bmjopen-2023-078134.
In recent years, discussions on the importance and scope of therapeutic value of new medicines have intensified, stimulated by the increase of prices and number of medicines entering the market. This study aims to perform a scoping review identifying factors contributing to the definition of the therapeutic value of medicines.
Scoping review.
We searched the MEDLINE, CINAHL, Embase, Business Source Premier, EconLit, Regional Business News, Cochrane, Web of Science, Scope and Pool databases through December 2020 in English, German, French, Italian and Spanish.
Studies that included determinants for the definition of therapeutic value of medicines were included.
Data were extracted using the mentioned data sources. Two reviewers independently screened and analysed the articles. Data were analysed from April 2021 to May 2022.
Of the 1883 studies screened, 51 were selected and the identified factors contributing to the definition of therapeutic value of medicines were classified in three categories: patient perspective, public health perspective and socioeconomic perspective. More than three-quarters of the included studies were published after 2014, with the majority of the studies focusing on either cancer disorders (14 of 51, 27.5%) or rare diseases (11 of 51, 21.6%). Frequently mentioned determinants for value were quality of life, therapeutic alternatives and side effects (all patient perspective), prevalence/incidence and clinical endpoints (all public health perspective), and costs (socioeconomic perspective).
Multiple determinants have been developed to define the therapeutic value of medicines, most of them focusing on cancer disorders and rare diseases. Considering the relevance of value of medicines to guide patients and physicians in decision-making as well as policymakers in resource allocation decisions, a development of evidence-based factors for the definition of therapeutic value of medicines is needed across all therapeutic areas.
近年来，随着进入市场的药品价格和数量的增加，人们对新药治疗价值的重要性和范围的讨论愈加强烈。本研究旨在进行范围综述，以确定影响药品治疗价值定义的因素。
范围综述。
我们用英语、德语、法语、意大利语和西班牙语在 MEDLINE、CINAHL、Embase、Business Source Premier、EconLit、Regional Business News、Cochrane、Web of Science、Scope 和 Pool 数据库中检索了截至 2020 年 12 月的文献。
纳入了关于药品治疗价值定义的决定因素的研究。
使用上述资料来源提取资料。两位评审员独立筛选和分析文章。资料分析于 2021 年 4 月至 2022 年 5 月进行。
在筛选出的 1883 项研究中，有 51 项被选中，确定的影响药品治疗价值定义的因素分为三类：患者视角、公共卫生视角和社会经济视角。超过四分之三的纳入研究是在 2014 年后发表的，其中大多数研究集中在癌症疾病（51 项研究中的 14 项，占 27.5%）或罕见疾病（51 项研究中的 11 项，占 21.6%）。经常提到的价值决定因素包括生活质量、治疗选择和副作用（均为患者视角）、流行率/发病率和临床终点（均为公共卫生视角）以及成本（社会经济视角）。
已经制定了多个决定因素来定义药品的治疗价值，其中大多数都集中在癌症疾病和罕见疾病上。考虑到药品治疗价值对指导患者和医生做出决策以及决策者在资源分配决策中的重要性，需要在所有治疗领域制定基于证据的治疗价值定义因素。
